EU approves Ultomiris for generalised myasthenia gravis
Ultomiris (ravulizumab), from Alexion Pharma/AstraZeneca, has been approved in Europe as an add-on to standard therapy for the treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive. This decision marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG in Europe. gMG is a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. The diagnosed prevalence of gMG in the EU is estimated at approximately 89,000
The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the CHAMPION-MG Phase III trial, which were published online in NEJM Evidence. In the trial, Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26, a patient-reported scale that assesses patients’ abilities to perform daily activities. Additionally, in prolonged follow-up results from the open-label extension, clinical benefit of Ultomiris was observed through 60 weeks.
In CHAMPION-MG, the safety profile of Ultomiris was comparable to placebo and consistent with that observed in Phase III trials of Ultomiris inejmevidence n paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). The most common adverse reactions in patients receiving Ultomiris were diarrhoea, upper respiratory tract infection, nasopharyngitis and headache.
See- "Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis"; Tuan Vu, M.D., Andreas Meisel, M.D., Renato Mantegazza, M.D., Djillali Annane, M.D., et al.,for the CHAMPION MG Study Group.Published April 26, 2022 . NEJM Evid 2022; 1 (5). DOI:https://doi.org/10.1056/EVIDoa2100066.
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